Retrophin, Inc. (OTCQB: RTRX) New York-based, biotechnology company focused on discovering and developing treatments for rare and life-threatening diseases. Retrophin is currently developing treatments for Focal Segmental Glomerulosclerosis (FSGS), Panto-thenate Kinase Associated Neurodegeneration (PKAN), Duchenne Muscular Dystrophy (DMD), and Spinal Muscular Atrophy (SMA).
Retrophin focuses on diseases that have no currently available treatments or very little competition. RTRX’s strategy focuses on "orphan drugs" because of the ability to get an expedited processing through the FDA, and such drugs can command a high price point once on the market. Orphan drug is a special designation that is given to drugs that are intended to treat very rare diseases (in the United States this means fewer than 200,000 patients).

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